JP2016528258A - 非コードrnaを形成するヘテロクロマチン - Google Patents

非コードrnaを形成するヘテロクロマチン Download PDF

Info

Publication number
JP2016528258A
JP2016528258A JP2016534865A JP2016534865A JP2016528258A JP 2016528258 A JP2016528258 A JP 2016528258A JP 2016534865 A JP2016534865 A JP 2016534865A JP 2016534865 A JP2016534865 A JP 2016534865A JP 2016528258 A JP2016528258 A JP 2016528258A
Authority
JP
Japan
Prior art keywords
oligonucleotide
gene
repeat
nucleotides
sequence
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2016534865A
Other languages
English (en)
Japanese (ja)
Inventor
ファティー オゾラック,
ファティー オゾラック,
Original Assignee
ラナ セラピューティクス インコーポレイテッド
ラナ セラピューティクス インコーポレイテッド
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by ラナ セラピューティクス インコーポレイテッド, ラナ セラピューティクス インコーポレイテッド filed Critical ラナ セラピューティクス インコーポレイテッド
Publication of JP2016528258A publication Critical patent/JP2016528258A/ja
Pending legal-status Critical Current

Links

Images

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • C12N2310/113Antisense targeting other non-coding nucleic acids, e.g. antagomirs
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering N.A.
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3231Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/341Gapmers, i.e. of the type ===---===
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/346Spatial arrangement of the modifications having a combination of backbone and sugar modifications

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Chemical & Material Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Neurosurgery (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2016534865A 2013-08-16 2014-08-15 非コードrnaを形成するヘテロクロマチン Pending JP2016528258A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361866894P 2013-08-16 2013-08-16
US61/866,894 2013-08-16
PCT/US2014/051257 WO2015023937A1 (fr) 2013-08-16 2014-08-15 Arn non codant formant de l'hétérochromatine

Publications (1)

Publication Number Publication Date
JP2016528258A true JP2016528258A (ja) 2016-09-15

Family

ID=52468720

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2016534865A Pending JP2016528258A (ja) 2013-08-16 2014-08-15 非コードrnaを形成するヘテロクロマチン

Country Status (6)

Country Link
US (1) US20150225722A1 (fr)
EP (1) EP3033114A4 (fr)
JP (1) JP2016528258A (fr)
CN (1) CN105682687A (fr)
CA (1) CA2921457A1 (fr)
WO (1) WO2015023937A1 (fr)

Cited By (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021532832A (ja) * 2018-08-02 2021-12-02 ダイン セラピューティクス, インコーポレーテッドDyne Therapeutics, Inc. フリートライヒ運動失調症を処置するための筋標的化複合体およびそれらの使用
US11844843B2 (en) 2021-07-09 2023-12-19 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US11911484B2 (en) 2018-08-02 2024-02-27 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
US11931421B2 (en) 2022-04-15 2024-03-19 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating myotonic dystrophy
US12012460B2 (en) 2018-08-02 2024-06-18 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
US12097263B2 (en) 2024-01-19 2024-09-24 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015023941A1 (fr) 2013-08-16 2015-02-19 Rana Therapeutics, Inc. Oligonucléotides ciblant des régions de l'euchromatine de gènes
EP3218483A4 (fr) 2014-11-14 2018-07-18 Ionis Pharmaceuticals, Inc. Composés et procédés de modulation des protéines
US12011488B2 (en) 2016-03-23 2024-06-18 The Regents Of The University Of California Methods of treating mitochondrial disorders
CA3018729A1 (fr) 2016-03-23 2017-09-28 The Regents Of The University Of California Methodes de traitement de troubles mitochondriaux
JP2019535839A (ja) 2016-11-29 2019-12-12 ピュアテック ヘルス エルエルシー 治療剤の送達のためのエクソソーム
WO2019126641A2 (fr) * 2017-12-21 2019-06-27 Ionis Pharmaceuticals, Inc. Modulation de l'expression de la frataxine
CA3171432A1 (fr) * 2020-03-16 2021-09-23 Stephanie CHERQUI Methodes de traitement de troubles mitochondriaux
IL313726A (en) * 2021-12-23 2024-08-01 Univ Massachusetts Medical treatment of fragile X-linked disorder

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE3329892A1 (de) * 1983-08-18 1985-03-07 Köster, Hubert, Prof. Dr., 2000 Hamburg Verfahren zur herstellung von oligonucleotiden
RU2377301C2 (ru) * 2003-12-23 2009-12-27 Сантарис Фарма А/С ОЛИГОМЕРНОЕ СОЕДИНЕНИЕ, ПОНИЖАЮЩЕЕ ЭКСПРЕССИЮ ЧЕЛОВЕЧЕСКОГО ГЕНА Bcl-2, КОНЪЮГАТ, ФАРМАЦЕВТИЧЕСКАЯ КОМПОЗИЦИЯ И ПРИМЕНЕНИЕ ОЛИГОМЕРНОГО СОЕДИНЕНИЯ ДЛЯ ЛЕЧЕНИЯ РАКА
WO2006063356A1 (fr) * 2004-12-10 2006-06-15 Isis Phamaceuticals, Inc. Regulation du controle epigenetique de l'expression genique
CN101501193B (zh) * 2006-08-11 2013-07-03 普罗森那技术公司 用于治疗与dna重复不稳定性相关的遗传病的方法和手段
WO2009090182A1 (fr) * 2008-01-14 2009-07-23 Santaris Pharma A/S Oligonucléotides de type « gapmère » de nucléotide d'adn substitué en c4'
CN101980726A (zh) * 2008-02-08 2011-02-23 普罗森那控股公司 治疗与dna重复不稳定性相关的遗传病症的方法和装置
KR20110091796A (ko) * 2008-12-04 2011-08-12 오피케이오 큐알엔에이, 엘엘씨 종양 억제 유전자에 대한 천연 안티센스 전사체의 억제에 의해 종양 억제 유전자 관련된 질환의 치료
GB201010557D0 (en) * 2010-06-23 2010-08-11 Mina Therapeutics Ltd RNA molecules and uses thereof
CN106434648A (zh) * 2010-07-19 2017-02-22 F·C·贝内特 肌强直性营养障碍蛋白激酶(dmpk)表达的调节
JP6188686B2 (ja) * 2011-06-09 2017-08-30 カッパーアールエヌエー,インコーポレイテッド フラタキシン(fxn)への天然アンチセンス転写物の阻害によるfxn関連疾患の治療
EP2751269B1 (fr) * 2011-08-29 2016-03-23 Ionis Pharmaceuticals, Inc. Procédés et composés utiles dans des pathologies associées à des expansions répétées
JP6129844B2 (ja) * 2011-09-14 2017-05-17 ラナ セラピューティクス インコーポレイテッド 多量体オリゴヌクレオチド化合物
US20160201064A1 (en) * 2013-08-16 2016-07-14 Rana Therapeutics, Inc. Compositions and methods for modulating expression of frataxin

Cited By (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021532832A (ja) * 2018-08-02 2021-12-02 ダイン セラピューティクス, インコーポレーテッドDyne Therapeutics, Inc. フリートライヒ運動失調症を処置するための筋標的化複合体およびそれらの使用
US11911484B2 (en) 2018-08-02 2024-02-27 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
US12012460B2 (en) 2018-08-02 2024-06-18 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
US11844843B2 (en) 2021-07-09 2023-12-19 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US11931421B2 (en) 2022-04-15 2024-03-19 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating myotonic dystrophy
US12097263B2 (en) 2024-01-19 2024-09-24 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy

Also Published As

Publication number Publication date
CA2921457A1 (fr) 2015-02-19
US20150225722A1 (en) 2015-08-13
CN105682687A (zh) 2016-06-15
EP3033114A4 (fr) 2017-04-05
EP3033114A1 (fr) 2016-06-22
WO2015023937A1 (fr) 2015-02-19

Similar Documents

Publication Publication Date Title
US10041074B2 (en) Euchromatic region targeting methods for modulating gene expression
EP2850190B1 (fr) Compositions et méthodes pour moduler l'expression de mecp2
JP2016528258A (ja) 非コードrnaを形成するヘテロクロマチン
EP2850186B1 (fr) Compositions et procédés de modulation de l'expression de la famille génique smn
JP6129844B2 (ja) 多量体オリゴヌクレオチド化合物
JP2015523853A (ja) Atp2a2発現を調節するための組成物及び方法
US20160201064A1 (en) Compositions and methods for modulating expression of frataxin
JP2015518713A (ja) Utrn発現を調節するための組成物及び方法
US20150232847A1 (en) Targeting oligonucleotides and related methods for modulating fxn rna
JP2015519057A (ja) Pten発現を調節するための組成物及び方法
JP2016521556A (ja) Foxp3発現を調節するための組成物及び方法
JP2015518710A (ja) ヘモグロビン遺伝子ファミリー発現を調節するための組成物及び方法
US20160201063A1 (en) Epigenetic regulators of frataxin
JP2015523855A (ja) Apoa1及びabca1発現を調節するための組成物及び方法
JP2015518711A (ja) Bdnf発現を調節するための組成物及び方法
US10758558B2 (en) Hybrid oligonucleotides and uses thereof
JP2014527819A5 (fr)
US20180030452A1 (en) Targeting oligonucleotides and uses thereof to modulate gene expression
AU2022203361A1 (en) Compositions and methods for modulating RNA